CA3058549A1 - Genetic construct for use in the treatment of neurodegenerative disorder or stroke - Google Patents

Genetic construct for use in the treatment of neurodegenerative disorder or stroke Download PDF

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Publication number
CA3058549A1
CA3058549A1 CA3058549A CA3058549A CA3058549A1 CA 3058549 A1 CA3058549 A1 CA 3058549A1 CA 3058549 A CA3058549 A CA 3058549A CA 3058549 A CA3058549 A CA 3058549A CA 3058549 A1 CA3058549 A1 CA 3058549A1
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CA
Canada
Prior art keywords
seq
genetic construct
use according
disease
set out
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CA3058549A
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English (en)
French (fr)
Inventor
Peter Widdowson
Keith Martin
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Quethera Ltd
Original Assignee
Quethera Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Quethera Ltd filed Critical Quethera Ltd
Publication of CA3058549A1 publication Critical patent/CA3058549A1/en
Pending legal-status Critical Current

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177Receptors; Cell surface antigens; Cell surface determinants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18Growth factors; Growth regulators
    • A61K38/185Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • A61P27/06Antiglaucoma agents or miotics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475Growth factors; Growth regulators
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/71Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Toxicology (AREA)
  • Psychology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
CA3058549A 2017-04-05 2018-03-28 Genetic construct for use in the treatment of neurodegenerative disorder or stroke Pending CA3058549A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB1705484.2 2017-04-05
GBGB1705484.2A GB201705484D0 (en) 2017-04-05 2017-04-05 Genetic construct
PCT/GB2018/050824 WO2018185468A1 (en) 2017-04-05 2018-03-28 Genetic construct for use in the treatment of neurodegenerative disorder or stroke

Publications (1)

Publication Number Publication Date
CA3058549A1 true CA3058549A1 (en) 2018-10-11

Family

ID=58682577

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3058549A Pending CA3058549A1 (en) 2017-04-05 2018-03-28 Genetic construct for use in the treatment of neurodegenerative disorder or stroke

Country Status (14)

Country Link
US (1) US20200046850A1 (pt)
EP (1) EP3606546B1 (pt)
JP (1) JP7296321B2 (pt)
KR (1) KR102616629B1 (pt)
CN (1) CN110809476B (pt)
AU (1) AU2018248651B2 (pt)
CA (1) CA3058549A1 (pt)
ES (1) ES2881176T3 (pt)
GB (1) GB201705484D0 (pt)
MX (1) MX2019012000A (pt)
PL (1) PL3606546T3 (pt)
PT (1) PT3606546T (pt)
RU (1) RU2757932C2 (pt)
WO (1) WO2018185468A1 (pt)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113454227A (zh) * 2018-12-19 2021-09-28 维萨梅布有限公司 编码蛋白质的rna
CN113557301A (zh) 2019-03-04 2021-10-26 公益财团法人东京都医学综合研究所 编码Trk片段的核酸构建体及其利用
AU2020248099A1 (en) * 2019-03-27 2021-10-07 Sigilon Therapeutics, Inc. Compositions, devices and methods for treating Fabry disease
GB202004832D0 (en) * 2020-04-01 2020-05-13 Instituto De Medicina Molecular Faculdade De Medicina Univ De Lisboa Therapeutic agaents, pharmaceutical compositions, and associated biomarkers
CN114933657B (zh) * 2021-08-25 2024-02-02 上海交通大学医学院 神经生长因子突变体重组蛋白及其应用
CN118256562A (zh) * 2022-12-26 2024-06-28 科辉智药(深圳)新药研究中心有限公司 一种用于中枢神经系统疾病治疗的基因序列构建体及其应用
WO2024161142A1 (en) 2023-02-02 2024-08-08 Quethera Limited Recombinant adeno-associated virus vector
EP4413993A1 (en) * 2023-02-10 2024-08-14 Dompe' Farmaceutici S.P.A. Method of obtaining recombinant human brain-derived neurotrophic factor

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2588221C (en) * 1999-06-08 2013-07-09 Regeneron Pharmaceuticals, Inc. Modified chimeric polypeptides with improved pharmacokinetic properties
US20030124095A1 (en) * 2001-12-31 2003-07-03 Regents Of The University Of California Methods for therapeutic use of brain derived neurotrophic factor in the entorhinal cortex
WO2009120978A2 (en) * 2008-03-27 2009-10-01 The Ohio State University Treatment of metabolic-related disorders using hypothalamic gene transfer of bdnf and compositions therfor
EP3165537A1 (en) * 2008-12-19 2017-05-10 H. Lundbeck A/S Modulation of the vps 10-domain receptor family for the treatment of mental and behavioural disorders
CN110438125A (zh) * 2012-03-15 2019-11-12 科纳公司 通过抑制脑源神经营养因子(bdnf)的天然反义转录物治疗bdnf相关疾病
GB2547179A (en) * 2015-10-26 2017-08-16 Quethera Ltd Genetic construct

Also Published As

Publication number Publication date
EP3606546A1 (en) 2020-02-12
AU2018248651B2 (en) 2024-07-25
KR102616629B1 (ko) 2023-12-21
WO2018185468A1 (en) 2018-10-11
CN110809476B (zh) 2024-02-27
JP2020516244A (ja) 2020-06-11
AU2018248651A1 (en) 2019-10-17
RU2757932C2 (ru) 2021-10-25
RU2019131098A3 (pt) 2021-07-27
PL3606546T3 (pl) 2021-11-08
ES2881176T3 (es) 2021-11-29
US20200046850A1 (en) 2020-02-13
CN110809476A (zh) 2020-02-18
MX2019012000A (es) 2020-01-27
RU2019131098A (ru) 2021-05-05
GB201705484D0 (en) 2017-05-17
EP3606546B1 (en) 2021-04-28
PT3606546T (pt) 2021-07-12
KR20200005549A (ko) 2020-01-15
JP7296321B2 (ja) 2023-06-22

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